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As non-inferiority was confirmed to assist interpretation of results, we also show the P values generated from tests of superiority (as recommended previously 30).
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"not superior" : Results of one study, evaluated as confirmatory, do not show statistically better efficacy (test of superiority: "superiority trial") in the primary endpoint in favour of one of the treatments.
The primary effectiveness end point will be evaluated for non-inferiority, with a nested test of superiority, based on the proportion of responders (defined as alive, free from HF-related events, with improvements in New York Heart Association class or improvement in Kansas City Cardiomyopathy Questionnaire quality of life score) at 12 months.
When the 95% interval excludes −5% but also lies above zero, a 2-sided P-value for the test of superiority was calculated.
No formal test of superiority was carried out for exploratory end points.
For any test of non-inferiority that is deemed significant, a subsequent test of superiority will be performed.
The treatment effect must remain in the model in order to substantiate the one-tailed test of superiority.
The P-values are only informational as they represent the variability in the estimated treatment effect rather than a formal test of superiority of DMF versus placebo.
By testing of superiority of XM02 versus placebo, assay sensitivity with respect to the primary endpoint, DSN in cycle 1, was confirmed.
(For comparison, a two-sided test of superiority indicates that that the minimum detectable effect size is 9.3 percentage points with 295 participants per group, assuming 75%% of participants in the food group have a MPR ≥95%%).
The guidance of the Committee for Proprietary Medicinal Products states that if the 95% CI for treatment effect lies above zero then there is evidence of statistical significance at the 5% level and it is acceptable to calculate the p value associated with a test of superiority [ 14].
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